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ASH 2025 | Exciting and potentially practice-changing hemophilia updates from ASH 2025
In this video, Jonathan Roberts, MD, The Bleeding & Clotting Disorders Institute, Peoria, IL, provides insight into promising therapeutic approaches for the treatment of hemophilia, highlighting some of the potentially practice-shaping updates presented at the ASH 2025 meeting. This interview took place at the 67th ASH Annual Meeting and Exposition, held in Orlando, FL.
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Transcript
I think there were a lot of good updates. You know, I think my colleague Dr. Malec’s study looking at the extended trial results for efanesoctocog alfa were encouraging. I think, you know, showing continued good bleed control with a high sustained factor VIII was very encouraging for the community, for those patients that are already on that medicine.
I think kind of the biggest, at least in my mind, landscape-changing potential treatments that are coming is the Mim8 and the NXT007 with a higher thrombin generation potential for factor VIII mimetic...
I think there were a lot of good updates. You know, I think my colleague Dr. Malec’s study looking at the extended trial results for efanesoctocog alfa were encouraging. I think, you know, showing continued good bleed control with a high sustained factor VIII was very encouraging for the community, for those patients that are already on that medicine.
I think kind of the biggest, at least in my mind, landscape-changing potential treatments that are coming is the Mim8 and the NXT007 with a higher thrombin generation potential for factor VIII mimetic. I think those drugs have the potential for those that want different delivery methodology, maybe less frequent dosing, yet normalizing hemostasis. I think those have the potential for practice-shaping advances. And so I think we will look forward to having more data in that regard.
Regarding gene therapy, I think that, you know, some of the final word on some of the gene therapy trials was given. I think we’re still, you know, the results are encouraging. I think the patient community is being a little more cautious in general with gene therapy. And so I think as more data continues to evolve, I think that will be more reassuring. And maybe even as we look towards new iterations of gene therapy, newer products, maybe more sustainable, more predictable, that those may get some more traction as far as clinical utility.
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